期刊
JOURNAL OF CYSTIC FIBROSIS
卷 19, 期 -, 页码 S19-S24出版社
ELSEVIER
DOI: 10.1016/j.jcf.2019.10.021
关键词
Membrane protein; ABC-transporter; Mutations; F508del; Therapy; Clinical drugs; Cystic fibrosis
资金
- Dutch Cystic Fibrosis Foundation, NCFS
- Netherlands Organization for Scientific Research (NWO) [731.017.420]
- Cystic Fibrosis Foundation Therapeutics (CFTR 3D Structure Consortium)
- French Association Vaincre La Mucoviscidose
- GENCI-[CINES] [2018-A0040707206, 2019-A0060707206]
- National Institutes of Health [NIHR01DK55835]
- Cystic Fibrosis Foundation
- Agence Nationale pour la Recherche
- Programme Hospitalier de Recherche Clinique
- Cystic Fibrosis Foundation Therapeutics grant [SHEPPA14XX0]
- Cystic Fibrosis Trust [SRC005/SRC011]
- Medical Research Council [MR/S00274X/1]
- MRC [MR/S00274X/1] Funding Source: UKRI
Structural biology and functional studies are a powerful combination to elucidate fundamental knowledge about the cystic fibrosis transmembrane conductance regulator (CFTR). Here, we discuss the latest findings, including how clinically-approved drugs restore function to mutant CFTR, leading to better clinical outcomes for people with cystic fibrosis (CF). Despite the prospect of regulatory approval of a CFTR-targeting therapy for most CF mutations, strenuous efforts are still needed to fully comprehend CFTR structure-and-function for the development of better drugs to enable people with CF to live full and active lives. (C) 2019 Published by Elsevier B.V. on behalf of European Cystic Fibrosis Society.
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